Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Case Reports
. 2018 Mar;65(3).
doi: 10.1002/pbc.26893. Epub 2017 Dec 12.

Response in a child with a BRAF V600E mutated desmoplastic infantile astrocytoma upon retreatment with vemurafenib

Cornelis M van Tilburg  1   2   3 , Florian Selt  1   2   3   4 , Felix Sahm  3   5   6 , Heidi Bächli  7 , Stefan M Pfister  1   2   3   8 , Olaf Witt  1   2   3   4 , Till Milde  1   2   3   4
Affiliations
Case Reports

Response in a child with a BRAF V600E mutated desmoplastic infantile astrocytoma upon retreatment with vemurafenib

Cornelis M van Tilburg et al. Pediatr Blood Cancer. 2018 Mar.

Abstract

Infants with low-grade glioma (LGG) and diencephalic syndrome have a poor outcome. The patient described here had a desmoplastic infantile astrocytoma harboring a BRAF V600E mutation. After relapse following initial standard chemotherapy treatment, he was successfully treated with the BRAF V600E inhibitor vemurafenib at the age of 3 years 11 months and 5 years 0 months. A rapid response was observed on both occasions. This illustrates the possibility of continuous oncogenic addiction and the therapeutic potential of BRAF V600E inhibitor monotherapy in LGG, even in very young severely compromised children. BRAF V600E inhibition in LGG and possible (re-)treatment regimens are briefly discussed.

Keywords: BRAF V600E inhibitor; child; desmoplastic infantile astrocytoma; infant; low-grade glioma; vemurafenib.

PubMed Disclaimer

Publication types

Supplementary concepts

LinkOut - more resources